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Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina

Abstract:

There is much debate on the adeno-associated virus (AAV) serotype that best targets specific retinal cell types and the route of surgical delivery — intravitreal or subretinal. This study compared three of the most efficacious AAV vectors known to date in a mouse model of retinal degeneration (rd1 mouse) and macaque and human retinal explants. GFP driven by a ubiquitous promoter was packaged into three AAV capsids: AAV2/8(Y733F), AAV2/2(quad Y-F) and AAV2/2(7m8). Overall AAV2/2(7m8) transduce...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Publisher's version

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Publisher copy:
10.1038/gt.2017.85

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Department:
Oxford, MSD, Clinical Neurosciences
Role:
Author
More by this author
Department:
Oxford, MSD, Clinical Neurosciences
Role:
Author
More by this author
Department:
Oxford, MSD, Clinical Neurosciences
Role:
Author
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Woolf Fisher Trust More from this funder
Wellcome Trust. More from this funder
Biotechnology and Biological Sciences Research Council More from this funder
National Institute for Health Research More from this funder
Medical Research Council More from this funder
Publisher:
Nature Publishing Group Publisher's website
Journal:
Gene Therapy Journal website
Volume:
24
Pages:
787–800
Publication date:
2017-11-05
Acceptance date:
2017-08-23
DOI:
EISSN:
1476-5462
ISSN:
0969-7128
Pubs id:
pubs:724038
URN:
uri:25a9b246-4079-469f-a7ac-01cd55a40f1c
UUID:
uuid:25a9b246-4079-469f-a7ac-01cd55a40f1c
Local pid:
pubs:724038

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